TOP Pharma & Biotech News #3?? March Edition

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In March, you'll read about the 8 key takeaways of the proposed FDA Modernization Act 3.0. Also, that molecular glues are the latest craze in pharma research, but what are they? Last, but not least read about the innovative chemotherapy approach that shows promise against lung cancer.

On Feb. 6, 2024, new legislation was introduced in the House of Representatives, called the FDA Modernization Act 3.0. This proposed legislation aims to reduce and replace the use of animals in nonclinical research, improve predictivity of nonclinical testing, and potentially reduce development times for drugs.

Background

Developmental drugs undergo preclinical trials in animals like rats, mice, beagles, and monkeys to assess safety and efficacy before human clinical trials. This stage faces criticism from animal welfare groups, who argue that drugs' responses in animals may not mirror those in humans, leading to a high failure rate in later human trials.

Key Points Of The Proposed FDA Modernization Act 3.0

1. Establishment of a nonclinical testing process — The proposed legislation directs the Secretary of Health and Human Services to establish a qualification process for nonclinical testing method, under which an entity/person may request qualification of a nonclinical testing method. The Secretary has the right to grant or deny such requests.
2. Eligibility for qualification — To be eligible for qualification, a nonclinical testing method must aim to replace or reduce animal testing and either improve the predictivity of nonclinical testing or reduce the development time for drugs.
3. Qualification process for nonclinical testing methods:Process to invite submission — The Secretary of HHS is to establish a process to invite submissions for the qualification of nonclinical testing methods.Guidance on submission content — At a minimum, the content should demonstrate the eligibility criteria: to replace or reduce animal testing and either improve the predictivity of nonclinical testing or reduce development time for drugs. Review of the submission — Using its senior managers and experienced staff, the FDA will review the information submitted and provide feedback. The FDA also can engage with external experts in its review of the submission.
4. Adjudication and timelines — Based on the information in the submission, along with information provided by FDA and external experts, the Secretary can accept or deny the request for a nonclinical testing method. The adjudication should take no more than 180 days from the date of submission.
5. Effect of successful qualification — If a nonclinical testing method is qualified, it becomes available for use in drug development within the specified context. The FDA will expedite the review of applications that use qualified methods and allow applicants to reference the qualification in their submissions.
6. Reporting — The Secretary is directed to publish annual reports on the FDA’s website evaluating the qualification process, including the types of methods qualified, the number of requests granted, review timelines, and the impact on animal use reduction.
7. Public meeting — Not later than 180 days after the legislation's enactment, the Secretary must announce a public meeting through the Federal Register. The purpose of this meeting is to gather input and recommendations from various stakeholders, including regulated industry, biomedical consortia, contract research organizations, and patients.
8. FDA guidance development — The Secretary of Health and Human Services, through the Commissioner of Food and Drugs, must propose guidance on the goals and implementation of the process through the proposed bill’s amendment to section 507A of the Federal Food, Drug, and Cosmetic Act within one year of the public meeting. This proposed guidance must then undergo a public comment period. The guidance must cover several key aspects, including the procedure for requesting qualification, eligibility criteria, information required from individuals seeking qualification, and the evaluation process that the Secretary will use for requests.

Please note that this legislation is not currently law and may not become a law unless it successfully proceeds through the law-making process.

Read more here .

Source: cell&gene

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Deals involving research into molecular glues have skyrocketed this year as big pharma bets on their revolutionary potential.

February’s announcement that Novo Nordisk was partnering with a biotech developing molecular glues to treat rare diseases was the latest in a series of expensive deals hoping to utilise the nascent technology.

Since their discovery in the early 1990s, molecular glues have become a promising areas of development in the oncology and rare diseases space, though “the potential breadth of […] applications is almost unlimited,” according to Sasso, Tenchov et al. The power of these small molecules lies in their ability to bind degradation enzymes to otherwise untargetable proteins by altering their shape, enabling targeted protein degradation (TPD).

Currently, the most popular molecular glues on the market are immunomodulatory imide drugs (IMiDs), a class made up of thalidomide and its less harmful analogues; notably lenalidomide, which saw over $6bn in sales in 2023, according to GlobalData.

GlobalData is the parent company of Pharmaceutical Technology.

The TPD market as a whole saw a 2,000% increase in the value of venture financing deals in 2022, but not all of this went to molecular glues. The other main category of TPDs are proteolysis-targeting chimeras, or PROTACs. These achieve a similar effect to the glues but act as a linking rather than a binding agent. This still creates the proximity required for the E3 enzyme to interact with the target protein but means that each PROTAC has to be designed with a specific protein in mind.

PROTACs are therefore comparatively less complex to manufacture, but also more limited in scope, as each one will typically only work for a clearly determined set of proteins as opposed to the “wide range of targets difficult to accurately predict a priori, exhibiting distinct biological activities,” of molecular glues.

In the same paper, Dong et al. report that “It is estimated that molecular glues could promote interactions between approximately 600 human E3 ligases and more than 20,000 potential human target proteins, providing a rich resource for exploring new targets and potential small-molecule drugs,” making the business case for investing in these drugs clear.

The recent shift in investment from PROTACs to molecular glues not only shows confidence in their potential but also reflects an increasing trend of huge spend on potential multiuse drugs. Novo Nordisk went from a relatively niche company focused on diabetes to the 13th largest company in the world on the back of its blockbuster GLP-1RA drugs, which are also proving to have a highly varied set of indications.

Another potential hope for new molecular glues comes in the form of artificial intelligence (AI). Historically, they have been discovered serendipitously, but advancements in automated drug discovery might enable a more rapid development by evaluating millions of potential formulations far faster than humans could. Bristol Myers Squibb is banking on this potential, partnering with tech startup VantAI to design and develop molecular glues to the tune of $674m just weeks before Novo’s deal.

Read more here .

Source: Pharmaceutical Technology

Lung cancer is not the most common form of cancer, but it is by far the deadliest.

Despite treatments such as surgery, radiation therapy and chemotherapy, only about a quarter of all people with the disease will live more than five years after diagnosis, and lung cancer kills more than 1.8 million people worldwide each year, according to the World Health Organization.

To improve the odds for patients with lung cancer, researchers from The University of Texas at Arlington and UT Southwestern Medical Center have pioneered a novel approach to deliver cancer-killing drugs directly into cancer cells.

"Our method uses the patient's own cellular material as a trojan horse to transport a targeted drug payload directly to the lung cancer cells," said Kytai T. Nguyen, lead author of a new study on the technique in the peer-reviewed Bioactive Materials and the Alfred R. and Janet H. Potvin Distinguished Professor in Bioengineering at UTA. "The process involves isolating T-cells (a type of immune cell) from the cancer patient and modifying them to express a specific receptor that targets the cancer cells."

The crucial step in this new technique involves isolating the cell membrane from these modified T-cells, loading the membranes with chemotherapy medications, and then coating them onto tiny drug-delivery granules. These nanoparticles are roughly 1/100 the size of a strand of hair.

When these membrane-coated nanoparticles are injected back into the patient, the cell membrane acts as a guide, directing the nanoparticles to the tumor cells with precision. This approach is designed to deceive the patient's immune system, as the coated nanoparticles mimic the properties of immune cells, avoiding detection and clearance by the body.

"The key advantage of this method lies in its highly targeted nature, which allows it to overcome the limitations of conventional chemotherapy that often lead to detrimental side effects and reduced quality of life for patients," said co-author Jon Weidanz, associate vice president for research and innovation and a researcher in kinesiology and bioengineering.

"By delivering chemotherapy directly to the tumor cells, the system aims to minimize collateral damage to healthy tissues," continued Weidanz, who also is a member of UTA’s Multi-Interprofessional Center for Health Informatics.

In the study, researchers loaded the nanoparticles with the anti-cancer drug Cisplatin. The membrane-coated nanoparticles accumulated in parts of the body with the tumors rather than in other parts of the body. As a result, this targeted delivery system was able to reduce the size of the tumors in the control group, demonstrating its efficacy.

"This personalized approach could pave the way for a new era of medicine tailored to each patient's unique characteristics and the specific nature of their tumor," Nguyen said. "The potential for reduced side effects and improved effectiveness makes our technique a noteworthy advancement in the field of cancer treatment."

Nguyen's work was supported by a $250,000 grant from the Cancer Prevention and Research Institute of Texas.

Read more here .

Source: WORLD PHARMA NEWS

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